Steroid Alternatives

Various studies looking at the impact of steroids, are now being published. They show that, overall, children with Duchenne who are treated with steroids, stay walking for longer than those who are not treated with steroids. The effect is variable from child to child, and the children who respond well to steroids, also seem to develop other complications less frequently. They develop breathing difficulties later, and have fewer problems with their spines. There may also be a positive effect on the heart muscle.

Prednisolone and Deflazacort are the steroids most often prescribed for Duchenne.

However, there can be important side effects, the worst of which are significant growth delay, cushingoid (puffy face), delayed puberty and a weakening of the bones. There is ongoing research to establish the most effective dose and regimens for steroids, to enable clinicians to minimise the side effects and maximise the benefits.

Potential alternatives to steroids

ReveraGen Biopharma, a US pharmaceutical company, is developing a new steroidal drug called Vamorolone (previously VBP15) which has the potential to better balance the benefit and side effects than the more traditional steroids. A successful Phase 1 study in adult volunteers was funded by Duchenne Children’s Trust, Joining Jack and the MDA (USA).

As of September 2016 ReveraGen Biopharma has begun recruiting patients for its Phase 2a open label study of Vamorolone. The trial will be recruiting patients who haven’t yet started steroids, between the ages of 4-7, and is recruiting in the UK, United States, Canada, Israel, Sweden and Australia.

CAT-1004 is being developed by the US company Catabasis. In animal models, the drug has shown to reduce muscle degeneration and improve muscle regeneration and function. The effects were seen across skeletal, cardiac and diaphragm muscle. CAT-1004 inhibits NF-ĸB, a protein that is activated in Duchenne as well as in other skeletal muscle disorders. CAT-1004 is not a steroid.

CAT-1004 is currently being tested on children with Duchenne who are not on steroids and are aged between 4-7 years in the phase 2 DMD-Move trial.

Taking part in a steroid alternative trial

All trials looking at steroid alternative treatments, will require participants to be “steroid naive”. This means they will not yet have started taking traditional steroids. These trials will most likely be recruiting younger patients.

Clinical stage

ReveraGen Biopharma

Vamorolone: long-term extension study

The steroid alternative is designed to treat patients with any DMD mutation.

The study is enrolling by invitation (participants who have taken part in the previous Vamorolone extension study). The study is enrolling 48 males between ages 4 and 7.

The 24-month phase II trial will take place across multiple sites, including some in the UK.

POLARIS DMD Trial. Phase 3 global trial for edasalonexent is currently being planned. Recruitment for this trial is not yet open. This trial is intended to support an application for commercial licensing of edasolonexent as a treatment for DMD.

Related

Nutraceuticals are products derived from food sources that provide extra health benefits, in addition to the basic nutritional value found in the food. Many nutraceuticals are thought to be beneficial to DMD.

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Duchenne affects approximately 1 in every 3,500 boys that are born but only around 1 in every 50 million girls. It may be rare, but it does happen. We have been speaking to Feriel, a 26 year old woman living with Duchenne muscular dystrophy. She has written us a short blog about her experiences with Duchenne from diagnosis to now.

Our co-founders Alex Johnson and Emily Crossley met after their sons were diagnosed with Duchenne muscular dystrophy. They both set up charities, Alex with Joining Jack, Emily with the Duchenne Children’s Trust.

We are proud to share with you our first ever Impact Report, read about the very real impact we are having and the many things we have been working on over the past six years to end Duchenne.
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